MILAN, Aug. 28, 2025(AAVantgarde), a clinical-stage biotechnology company developing next-generation gene therapies for inherited retinal diseases, today announced the presentation of updated LUCE-1 clinical data at the 25th European Society of Retina Specialists (EURETINA) Annual Congress (EURetina 2025), taking place in Paris 4-7 September 2025. AAVantgarde will also be presenting at the Ophthalmology Futures Forum (OFF) and EURetina Innovation Spotlight (EIS) events on the 3rd September in Paris.
The oral presentation from the LUCE-1 clinical trial builds on the data presented at the FLORetina medical conference in December 2024 and ARVO in May 2025, and will be presented by LUCE-1's Principal Investigator, Prof. Francesca Simonelli, Head of the Ophthalmology Unit at the University Hospital of Campania "Luigi Vanvitelli" (Naples), who will highlight the most recent clinical data from this study, designed to evaluate the safety and efficacy of AAVantgarde's dual hybrid AAV-based gene therapy for patients with retinitis pigmentosa secondary to Usher 1B, caused by mutations in the MYO7A gene.
Oral Presentation details:
Presentation Number: O13
Abstract Title: LUCE: A first-in-human Phase 1/2 multicenter, dose-escalation, safety and efficacy study of subretinal administration of dual-hybrid AAVB-081 gene therapy in subjects with Usher Syndrome type 1B Retinitis Pigmentosa
Session Title: Free Paper 2 - Inherited Retinal Diseases
Session Date/Time: May 4, 2025, from 12:00 to 13:00 h CEST
AAVantgarde will also be available for partnering and scientific discussions throughout the congress.
About AAVantgarde
AAVantgarde Bio is a clinical stage, biotechnology company advancing best-in-class therapies for patients with inherited retinal diseases. The company's lead programs target Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B, two severe, inherited retinal diseases with no approved treatments. AAVB-039 and AAVB-081 are investigational, dual AAV gene therapies designed to address the root genetic causes of these diseases. With a strong foundation in translational science and a commitment to clinical excellence, AAVantgarde is working to bring transformative therapies to patients. For more information, please visit: www.aavantgarde.com
About the LUCE-1 Trial
About Usher syndrome type 1B
Usher syndrome type 1B (Usher1B) is an inherited disease that affects the retina and the inner ear. Usher1B is caused by mutations in the MYO7A gene. The therapeutic gene to treat Usher1B is 6.7 kb long and is therefore too large to fit inside a standard AAV vector. Approximately 20,000 patients in the U.S. and E.U. have Usher1B. These children are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life. Although there are surgical treatments available to treat deafness in these patients, there are no treatments available to treat progressive vision loss and blindness in these patients.
Contact:
Dr. Magda Blanco - Head of Corporate Development
Email: info@aavantgarde.com
